Gene therapy achieves early success in hemophilia

Symptoms improved significantly in adults with the hereditary bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St Jude Children's Research Hospital in Memphis, USA, and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the UK.

The findings of the six-person study mark the first proof that gene therapy can reduce disabling, painful bleeding episodes in patients with the inherited blood disorder. Results of the Phase I study appear in today's online edition of the New England Journal of Medicine. The research was also presented at the 53rd annual meeting of the American Society of Hematology in San Diego.

Four study participants stopped receiving protein injections to prevent bleeding episodes after undergoing the therapy and have not suffered spontaneous bleeding. Several have also participated in marathons and other activities that would have been difficult prior to gene therapy. The study volunteers were all treated at the Royal Free Hospital in London under the care of Edward Tuddenham, a pioneer in the field of blood coagulation and a study co-author.